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Sarepta Therapeutics’ CEO Chris Garabedian Is Interviewed

January 9, 2013

       The Muscular Dystrophy Association (MDA) publishes online on January 7, 2013, an article titled, “Answers to the Critical Questions About Eteplirsen for DMD”. Chris Garabedian, President and CEO of Sarepta Therapeutics, has a Q&A session with MDA to identify the next steps for the orphan drug Eteplirsen for Duchenne Muscular Dystrophy (DMD).

The following are a few points made by Mr. Garabedian :

1) Plans to meet with FDA in 1st Q2013 to review Phase 2b trial data

2) Several registration paths (i.e. accelerated approval, traditional Phase 3 Study) available, including a new regulatory advance for rare diseases, PDUFA V(5), which is signed into law in 2012

3) Currently working to “scale up manufacturing of Eteplirsen to support future clinical development …. We have a plan in place to meet this challenge …..”

4) Researching additional exon-skipping compounds that target exon 45, 50, & 53.


**** 1) January 9, 2013, 4:30 p.m. PT, Wednesday – Company Overview Presentation at the 31st J.P. Morgan Global Healthcare Conference. Chris Garabedian, Sarepta’s President and CEO, will be the presenter. The presentation will be webcast live under the events section of Sarepta Therapeutics’ website

2) Plans to ask for a meeting with the FDA for scheduling a meeting for the 1st Quarter of 2013 to discuss asking for Accelerated Approval for Eteplirsen and to also discuss a Phase III clinical trial for Eteplirsen

3) Investors probably will not be hearing about the results of the FDA meeting until about March – April 2013

4) Plans are for a future meeting with the FDA about the chemistry, manufacturing, and controls (CMC)

5) 74-week Eteplirsen clinical trial data.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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